Trial Outcomes & Findings for A Phase I, Multicenter, Dose Escalation Study of CAT-8015 in Participants With Chronic Leukemia (NCT NCT00587457)

Last Updated: 2017-07-06

Results Overview

An adverse event (AE) events present at baseline that worsened in intensity after administration of investigational product or events absent at baseline that emerged after administration of study drug, for the period extending to 30 days after the last dose of study drug. A serious adverse event (SAE) is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening situation (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly/birth defect in the offspring of a participant who received moxetumomab pasudotox. Treatment-emergent were events between administration of investigational product and Day 28 that were absent before treatment or that worsened relative to pretreatment state.

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

11 participants

Primary outcome timeframe

From start of study drug administration until 30 days after the last dose of study drug

Results posted on

2017-07-06

Participant Flow

A total of 11 participants were enrolled, of which all participants discontinued from the treatment.

Participant milestones

Participant milestones
Measure	CAT-8015 5 Microgram Per Kilogram (mcg/kg) Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 10 Microgram Per Kilogram (mcg/kg) Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 20 Microgram Per Kilogram (mcg/kg) Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Overall Study STARTED	3	3	5
Overall Study COMPLETED	0	0	0
Overall Study NOT COMPLETED	3	3	5

Reasons for withdrawal

Reasons for withdrawal
Measure	CAT-8015 5 Microgram Per Kilogram (mcg/kg) Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 10 Microgram Per Kilogram (mcg/kg) Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 20 Microgram Per Kilogram (mcg/kg) Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Overall Study Adverse Event	1	2	1
Overall Study Disease progression	2	0	2
Overall Study Initiation of alternative therapy	0	1	0
Overall Study Undefined	0	0	1
Overall Study Development of neutralizing Abs	0	0	1

Baseline Characteristics

A Phase I, Multicenter, Dose Escalation Study of CAT-8015 in Participants With Chronic Leukemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CAT-8015 5 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 10 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 20 Microgram Per Kilogram (mcg/kg) n=5 Participants Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	Total n=11 Participants Total of all reporting groups
Age, Continuous	58.7 Years STANDARD_DEVIATION 2.3 • n=99 Participants	62.0 Years STANDARD_DEVIATION 4.4 • n=107 Participants	61.6 Years STANDARD_DEVIATION 7.5 • n=206 Participants	60.9 Years STANDARD_DEVIATION 5.4 • n=7 Participants
Sex: Female, Male Female	1 Participants n=99 Participants	1 Participants n=107 Participants	1 Participants n=206 Participants	3 Participants n=7 Participants
Sex: Female, Male Male	2 Participants n=99 Participants	2 Participants n=107 Participants	4 Participants n=206 Participants	8 Participants n=7 Participants

PRIMARY outcome

Timeframe: From start of study drug administration until 30 days after the last dose of study drug

Population: Safety population included all participants who received any treatment of moxetumomab pasudotox.

Outcome measures

Outcome measures
Measure	CAT-8015 5 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 10 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 20 Microgram Per Kilogram (mcg/kg) n=5 Participants Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) TEAEs	3 Participants	3 Participants	5 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) TESAEs	1 Participants	2 Participants	3 Participants

PRIMARY outcome

Timeframe: From start of study drug administration until 30 days after the last dose of study drug

Population: Safety population included all participants who received any treatment of moxetumomab pasudotox.

The vital sign abnormalities which require an action or intervention by the investigator, or a finding judged by the investigator were reported as an adverse event. TEAEs were events present at baseline that worsened in intensity after administration of study drug or events absent at baseline that emerged after administration of study drug, for the period extending to 30 days after the last dose of study drug.

Outcome measures

Outcome measures
Measure	CAT-8015 5 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 10 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 20 Microgram Per Kilogram (mcg/kg) n=5 Participants Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Number of Participants With Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Pyrexia	1 Participants 0.56	0 Participants 0.70	1 Participants 0.29
Number of Participants With Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Sinus tachycardia	1 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: From start of study drug administration until 30 days after the last dose of study drug

Population: Safety population included all participants who received any treatment of moxetumomab pasudotox.

AEs observed in participants with clinically significant ECG abnormalities were assessed.

Outcome measures

Outcome measures
Measure	CAT-8015 5 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 10 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 20 Microgram Per Kilogram (mcg/kg) n=5 Participants Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Number of Participants With Clinically Relevant Electrocardiogram (ECG) Abnormalities Recorded as Adverse Events (AEs)	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: From start of study drug administration until 30 days after the last dose of study drug

Population: Safety population included all participants who received any treatment of moxetumomab pasudotox.

An abnormal laboratory finding which required an action or intervention by the investigator, or a finding judged by the investigator to represent a change beyond the range of normal physiologic fluctuation were reported as an adverse event. Treatment-emergent were events between first dose of study drug and 30 days after the last dose that were absent before treatment or that worsened relative to pretreatment state. Number of participants with grade 3 or higher treatment-emergent adverse events for laboratory abnormalities were reported as clinically relevant laboratory changes.

Outcome measures

Outcome measures
Measure	CAT-8015 5 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 10 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 20 Microgram Per Kilogram (mcg/kg) n=5 Participants Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Hemoglobin decreased	3 Participants	0 Participants	1 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Platelet count decreased	2 Participants	1 Participants	1 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Neutrophil count decreased	2 Participants	0 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Lymphocyte count decreased	1 Participants	0 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) White blood cell count decreased	1 Participants	0 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Neutropenia	1 Participants	0 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Febrile neutropenia	0 Participants	1 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Blood albumin decreased	1 Participants	1 Participants	2 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Blood magnesium decreased	1 Participants	2 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Blood glucose increased	2 Participants	0 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Blood magnesium increased	1 Participants	1 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Blood phosphorus decreased	1 Participants	1 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Aspartate aminotransferase increased	0 Participants	1 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Blood glucose decreased	0 Participants	0 Participants	1 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Blood potassium decreased	0 Participants	1 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Blood sodium decreased	1 Participants	0 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Blood sodium increased	0 Participants	1 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Blood triglycerides increased	1 Participants	0 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Blood alkaline phosphatase increased	0 Participants	1 Participants	0 Participants
Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs) Blood calcium increased	0 Participants	1 Participants	0 Participants

PRIMARY outcome

Timeframe: Up to 2 years of post-treatment follow-up

Population: Safety population included all participants who received any treatment of moxetumomab pasudotox.

Objective response rate defined as the proportion of participants with confirmed complete response (CR) or confirmed partial response (PR) according to Response Evaluation Criteria.

Outcome measures

Outcome measures
Measure	CAT-8015 5 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 10 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 20 Microgram Per Kilogram (mcg/kg) n=5 Participants Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Number of Participants With Objective Response Rate (ORR): Complete Response (CR) or Partial Response (PR)	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Up to 2 years of post-treatment follow-up

Population: Safety population included all participants who received any treatment of moxetumomab pasudotox.

Antitumor activity was assessed by best overall objective tumor response.

Outcome measures

Outcome measures
Measure	CAT-8015 5 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 10 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 20 Microgram Per Kilogram (mcg/kg) n=5 Participants Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Best Overall Objective Tumor Response Complete response (CR)	0 Participants	0 Participants	0 Participants
Best Overall Objective Tumor Response Partial Response (PR)	0 Participants	0 Participants	0 Participants
Best Overall Objective Tumor Response Stable Disease (SD)	2 Participants	3 Participants	2 Participants
Best Overall Objective Tumor Response Progressive Disease (PD)	1 Participants	0 Participants	3 Participants

PRIMARY outcome

Timeframe: Predose, 0.25 (During Infusion), 0.5 (End of Infusion), 1, 1.5, 2, 4, 8 and 12 hours postdose on Day 1 and 5; Predose and End of Infusion on Day 3

Population: Safety population included all participants who received any treatment of moxetumomab pasudotox. Here 'n' signifies participants evaluable for specified categories, for each arm, respectively.

The Tmax is the time to reach maximum observed plasma concentration of Moxetumomab Pasudotox.

Outcome measures

Outcome measures
Measure	CAT-8015 5 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 10 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 20 Microgram Per Kilogram (mcg/kg) n=5 Participants Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox Cycle 1, Day 1 (n=3,3,5)	0.500 hour Interval 0.5 to 0.5	0.50 hour Interval 0.5 to 0.5	0.500 hour Interval 0.0 to 0.5
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox Cycle 1, Day 5 (n=3,3,5)	0.500 hour Interval 0.5 to 0.5	0.500 hour Interval 0.3 to 0.5	0.500 hour Interval 0.3 to 1.0
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox Cycle 2, Day 1 (n=2,2,2)	4.25 hour Interval 0.5 to 8.0	0.375 hour Interval 0.25 to 0.5	0.500 hour Interval 0.5 to 0.5
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox Cycle 2, Day 5 (n=2,2,2)	0.500 hour Participant had no measurable Tmax concentration.	0.800 hour Interval 0.5 to 1.0	0.500 hour Interval 0.5 to 0.5
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox Cycle 3, Day 1 (n=1,1,2)	0.500 hour Median range was not evaluable since only 1 participant was evaluated.	0.500 hour Median range was not evaluable since only 1 participant was evaluated.	0.500 hour Interval 0.5 to 0.5
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox Cycle 3, Day 5 (n=1,1,2)	0.500 hour Median range was not evaluable since only 1 participant was evaluated.	2.00 hour Median range was not evaluable since only 1 participant was evaluated.	0.500 hour Interval 0.5 to 0.5
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox Cycle 4, Day 1 (n=1,NA,NA)	0.500 hour Median range was not evaluable since only 1 participant was evaluated.	NA hour Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.	NA hour Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox Cycle 4, Day 5 (n=1,NA,NA)	0.500 hour Median range was not evaluable since only 1 participant was evaluated.	NA hour Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.	NA hour Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox Cycle 5, Day 1 (n=1,NA,NA)	0.500 hour Median range was not evaluable since only 1 participant was evaluated.	NA hour Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.	NA hour Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox Cycle 5, Day 5 (n=1,NA,NA)	0.500 hour Median range was not evaluable since only 1 participant was evaluated.	NA hour Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.	NA hour Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox Cycle 6, Day 1 (n=1,NA,NA)	0.500 hour Median range was not evaluable since only 1 participant was evaluated.	NA hour Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.	NA hour Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Moxetumomab Pasudotox Cycle 6, Day 5 (n=1,NA,NA)	0.500 hour Median range was not evaluable since only 1 participant was evaluated.	NA hour Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.	NA hour Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.

PRIMARY outcome

Timeframe: Predose, 0.25 (During Infusion), 0.5 (End of Infusion), 1, 1.5, 2, 4, 8 and 12 hours postdose on Day 1 and 5; Predose and End of Infusion on Day 3

The Cmax is the maximum observed plasma concentration of Moxetumomab Pasudotox.

Outcome measures

Outcome measures
Measure	CAT-8015 5 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 10 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 20 Microgram Per Kilogram (mcg/kg) n=5 Participants Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox Cycle 6, Day 1 (n=1,NA,NA)	93.2 nanogram per milliliter Median range was not evaluable since only 1 participant was evaluated.	NA nanogram per milliliter Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.	NA nanogram per milliliter Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox Cycle 1, Day 1 (n=3,3,5)	97 nanogram per milliliter Interval 59.1 to 107.0	57.7 nanogram per milliliter Interval 46.5 to 145.0	180 nanogram per milliliter Interval 63.7 to 351.0
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox Cycle 1, Day 5 (n=3,3,5)	80.9 nanogram per milliliter Interval 56.4 to 122.0	117 nanogram per milliliter Interval 44.7 to 166.0	206 nanogram per milliliter Interval 108.0 to 353.0
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox Cycle 2, Day 1 (n=2,2,2)	94.0 nanogram per milliliter Interval 66.0 to 122.0	723 nanogram per milliliter Interval 146.0 to 1300.0	156 nanogram per milliliter Interval 134.0 to 178.0
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox Cycle 2, Day 5 (n=2,2,2)	50.5 nanogram per milliliter Interval 0.0 to 101.0	80.8 nanogram per milliliter Interval 72.3 to 89.2	211 nanogram per milliliter Interval 171.0 to 250.0
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox Cycle 3, Day 1 (n=1,1,2)	107 nanogram per milliliter Median range was not evaluable since only 1 participant was evaluated.	154 nanogram per milliliter Median range was not evaluable since only 1 participant was evaluated.	160 nanogram per milliliter Interval 63.9 to 257.0
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox Cycle 3, Day 5 (n=1,1,2)	93.4 nanogram per milliliter Median range was not evaluable since only 1 participant was evaluated.	200 nanogram per milliliter Median range was not evaluable since only 1 participant was evaluated.	266 nanogram per milliliter Interval 156.0 to 376.0
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox Cycle 4, Day 1 (n=1,NA,NA)	84.9 nanogram per milliliter Median range was not evaluable since only 1 participant was evaluated.	NA nanogram per milliliter Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.	NA nanogram per milliliter Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox Cycle 4, Day 5 (n=1,NA,NA)	86.2 nanogram per milliliter Median range was not evaluable since only 1 participant was evaluated.	NA nanogram per milliliter Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.	NA nanogram per milliliter Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox Cycle 5, Day 1 (n=1,NA,NA)	95.0 nanogram per milliliter Median range was not evaluable since only 1 participant was evaluated.	NA nanogram per milliliter Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.	NA nanogram per milliliter Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox Cycle 5, Day 5 (n=1,NA,NA)	86.9 nanogram per milliliter Median range was not evaluable since only 1 participant was evaluated.	NA nanogram per milliliter Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.	NA nanogram per milliliter Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.
Maximum Observed Serum Concentration (Cmax) for Moxetumomab Pasudotox Cycle 6, Day 5 (n=1,NA,NA)	67.0 nanogram per milliliter Median range was not evaluable since only 1 participant was evaluated.	NA nanogram per milliliter Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.	NA nanogram per milliliter Data was not evaluated due to the small size of the sample cohorts and limited quantifiable data collected.

SECONDARY outcome

Timeframe: Up to end of treatment (4-6 weeks after the last dose)

Population: Safety population included all participants who received any treatment of moxetumomab pasudotox.

Participants tested for immunogenicity to moxetumomab pasudotox prior to enrollment, before each cycle and at end of study. The neutralization assay measures the capacity of participant's plasma (antibodies) to inhibit the binding of moxetumomab pasudotox to its target, cluster of differentiation 22 (CD22), coated onto enzyme linked immunosorbent assay (ELISA) plates. It was used as a direct surrogate for biological activity based on the mechanism of action of this drug. Significant level of neutralizing antibody activity defined as the capacity of test plasma to inhibit greater than (\>)50 percentage (%) of the binding of CAT-8015 to CD22 using an ELISA-based method.

Outcome measures

Outcome measures
Measure	CAT-8015 5 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 10 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 20 Microgram Per Kilogram (mcg/kg) n=5 Participants Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Number of Participants With Positive Neutralizing Antibodies	0 Participants	0 Participants	1 Participants

SECONDARY outcome

Timeframe: End of treatment (4-6 weeks after the last dose)

Population: Safety population included all participants who received any treatment of moxetumomab pasudotox.

Participants demonstrated CD22 expression on malignant cells at Screening and CD22 is a regulatory molecule that prevents the over activation of the immune system and the development of autoimmune diseases.

Outcome measures

Outcome measures
Measure	CAT-8015 5 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 10 Microgram Per Kilogram (mcg/kg) n=3 Participants Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 20 Microgram Per Kilogram (mcg/kg) n=5 Participants Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Percentage Change From Baseline in Cluster of Differentiation 22 (CD22) Expression (MESF CD22-PE [of CD5+/CD19+])	1139 Percentage Standard Deviation 1514.6	-3076 Percentage Standard Deviation 5982.1	-342.8 Percentage Standard Deviation 1816.8
Percentage Change From Baseline in Cluster of Differentiation 22 (CD22) Expression CD22-PE [OF CD5+/CD19+]	-33.65 Percentage Standard Deviation 27.79	-10.25 Percentage Standard Deviation 16.05	-8.85 Percentage Standard Deviation 16.19

Adverse Events

CAT-8015 5 Microgram Per Kilogram (mcg/kg)

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

CAT-8015 10 Microgram Per Kilogram (mcg/kg)

Serious events: 2 serious events

Other events: 3 other events

Deaths: 0 deaths

CAT-8015 20 Microgram Per Kilogram (mcg/kg)

Serious events: 3 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	CAT-8015 5 Microgram Per Kilogram (mcg/kg) n=3 participants at risk Participants received a single intravenous infusion of 5 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 10 Microgram Per Kilogram (mcg/kg) n=3 participants at risk Participants received a single intravenous infusion of 10 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.	CAT-8015 20 Microgram Per Kilogram (mcg/kg) n=5 participants at risk Participants received a single intravenous infusion of 20 mcg/kg moxetumomab pasudotox (CAT-8015) on Days 1, 3, and 5 of every 28-day cycle and continued cycles of therapy until progressive disease (PD) or until otherwise they become ineligible.
Blood and lymphatic system disorders Febrile neutropenia	0.00% 0/3 • From start of study drug administration until 30 days after the last dose of study drug	33.3% 1/3 • Number of events 1 • From start of study drug administration until 30 days after the last dose of study drug	0.00% 0/5 • From start of study drug administration until 30 days after the last dose of study drug
Eye disorders Optic ischaemic neuropathy	0.00% 0/3 • From start of study drug administration until 30 days after the last dose of study drug	33.3% 1/3 • Number of events 1 • From start of study drug administration until 30 days after the last dose of study drug	0.00% 0/5 • From start of study drug administration until 30 days after the last dose of study drug
Infections and infestations Acute pulmonary histoplasmosis	0.00% 0/3 • From start of study drug administration until 30 days after the last dose of study drug	33.3% 1/3 • Number of events 1 • From start of study drug administration until 30 days after the last dose of study drug	0.00% 0/5 • From start of study drug administration until 30 days after the last dose of study drug
Infections and infestations Infection	33.3% 1/3 • Number of events 1 • From start of study drug administration until 30 days after the last dose of study drug	33.3% 1/3 • Number of events 1 • From start of study drug administration until 30 days after the last dose of study drug	0.00% 0/5 • From start of study drug administration until 30 days after the last dose of study drug
Infections and infestations Neutropenic infection	0.00% 0/3 • From start of study drug administration until 30 days after the last dose of study drug	33.3% 1/3 • Number of events 1 • From start of study drug administration until 30 days after the last dose of study drug	0.00% 0/5 • From start of study drug administration until 30 days after the last dose of study drug
Infections and infestations Pneumonia	33.3% 1/3 • Number of events 1 • From start of study drug administration until 30 days after the last dose of study drug	0.00% 0/3 • From start of study drug administration until 30 days after the last dose of study drug	20.0% 1/5 • Number of events 1 • From start of study drug administration until 30 days after the last dose of study drug
Investigations Blood calcium increased	0.00% 0/3 • From start of study drug administration until 30 days after the last dose of study drug	33.3% 1/3 • Number of events 1 • From start of study drug administration until 30 days after the last dose of study drug	0.00% 0/5 • From start of study drug administration until 30 days after the last dose of study drug
Metabolism and nutrition disorders Tumour lysis syndrome	0.00% 0/3 • From start of study drug administration until 30 days after the last dose of study drug	0.00% 0/3 • From start of study drug administration until 30 days after the last dose of study drug	20.0% 1/5 • Number of events 1 • From start of study drug administration until 30 days after the last dose of study drug
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/3 • From start of study drug administration until 30 days after the last dose of study drug	0.00% 0/3 • From start of study drug administration until 30 days after the last dose of study drug	20.0% 1/5 • Number of events 1 • From start of study drug administration until 30 days after the last dose of study drug
Respiratory, thoracic and mediastinal disorders Obstructive airways disorder	0.00% 0/3 • From start of study drug administration until 30 days after the last dose of study drug	0.00% 0/3 • From start of study drug administration until 30 days after the last dose of study drug	20.0% 1/5 • Number of events 1 • From start of study drug administration until 30 days after the last dose of study drug

Other adverse events

Additional Information

Mark C Lanasa

MedImmune, LLC.

Phone: 301-398-0000

Email: [email protected]

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